OncoMatch/Clinical Trials/NCT06449001

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis

Is NCT06449001 recruiting? Yes, currently enrolling (May 2026). This Phase 3 trial studies Danicopan for paroxysmal nocturnal hemoglobinuria.

Phase 3RecruitingAlexion Pharmaceuticals, Inc.NCT06449001Data as of May 2026

Treatment: Danicopan — The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).

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Extracted eligibility criteria

Prior therapy

Must have received: C5 inhibitor (ravulizumab, eculizumab)

Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study.

Lab requirements

Blood counts

Platelet count ≥ 30000/μL and no need for platelet transfusions; ANC ≥ 500/μL

Liver function

ALT ≤ 2 × ULN or ≤ 3 × ULN for participants with documented liver iron overload (serum ferritin ≥ 500 ng/mL); Direct bilirubin ≤ 2 × ULN unless due to hemolysis or Gilbert's syndrome

Platelet count < 30000/μL or there is a need for platelet transfusions. ANC < 500/μL. Clinically significant laboratory abnormalities related to liver function, including: ALT > 2 × ULN or ALT > 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL. Direct bilirubin > 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history.

Structured fields extracted by AI. May contain errors — verify against the official protocol.

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